A Phase 1 and pharmacokinetic study of Uproleselan (GMI-1271, IND #139758, NSC #801708) in combination with fludarabine and cytarabine for patients with acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that expresses E-selectin ligand on the cell membrane and is in second or greater relapse or that is refractory to relapse therapy

This study is being done to see how safe an investigational drug is and how well it will work to help people with obesity, or overweight with weight-related conditions like hypertension, dyslipidemia, obstructive sleep apnea, non-alcoholic fatty liver disease, or diabetes. If you qualify to be in the study, you will be given frequent lifestyle and behavioral counseling for the first 12 weeks of the study. The counseling will consist of advice on physical activities and dietary advice on healthy eating. During the treatment period, you will receive either tirzepatide or placebo. Placebo is a solution that looks like the study drug but has no medicine. The chance that you will get the study drug is 2 in 3. This phase will last about 72 weeks.

The purpose of this multicenter study is to compare outcomes between patients who will undergo different types of ACL reconstruction. All patients will have a tendon from their own knee used to reconstruct the ACL. Prior to knee surgery, researchers will randomize (i.e. a coin toss) to determine if ACL reconstruction will be done with patellar or quadriceps tendon and if the lateral extra-articular tenodesis will or will not be added to the ACL surgery. We will follow-up with participants as they undergo treatment and recovery after surgery for 2 years.

TrialNet is an international research group dedicated to the study, prevention, and early treatment of Type 1 diabetes. Type 1 diabetes is now understood as a disease that develops over time in stages. Stage 1 starts with the appearance of having two or more autoantibodies. This is followed by Stage 2, which is the development of abnormal blood glucose levels. Stage 3 is the clinical diagnosis of Type 1 diabetes. This study will help us learn more about how Type 1 diabetes occurs through the screening of diabetes-related autoantibodies and monitoring individuals who have tested positive for these autoantibodies.

The goal of this study is to support the validation of a revision to the Autism Diagnostic Interview – Revised (ADI-R). The ADI-R is a highly validated diagnostic tool used in clinical assessments for autism spectrum disorder and widely used in research for participant characterization and inclusion. Aims: 1. To explore the validity of the ADI-3 (concurrent validity with ADI-R and other autism instruments; predictive validity with best estimate clinical diagnosis). 2. To explore the reliability of the ADI-3 (interrater, test-retest, internal consistency) 3. To derive diagnostic algorithms that better match DSM-5 criteria and maximize sensitivity and specificity 4. To ensure that changes (e.g., reduced length, new format to codes) yield sufficient information to inform diagnoses.

This study is enrolling participants who have been diagnosed with juvenile spondyloarthritis, are taking a tumor necrosis factor inhibitor (TNFi) and have reached a clinically inactive disease state for a minimum of six months. Researchers want to know if children who have maintained inactive disease for at least 6 months can maintain quiet disease without taking their medication as frequently or stop the TNFi therapy. Quiet disease means that disease related symptoms are not active or being experienced in the patient. Researchers also want to know the safest method to bring patients off medication. If a flare does occur during therapy reduction, researchers want to find out whether they can predict when a flare is most likely to happen, and how quickly an inactive disease state can be recaptured.

This study is being done to look at how pregnancy affects the health of women with cystic fibrosis (CF) and to look at the health of babies born to women with CF. It is expected that most but not all women will be taking the newest CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator medication called TRIKAFTA®. It is not known how this medication will affect the health of mother and infant.

The purpose of this study is to learn whether the use of Pregnyl with the drug ruxolitinib is able to reduce the need for high dose steroids to treat severe acute Graft versus Host Disease (GVHD).

University of Minnesota researchers are conducting a research study to learn more about how an individually-delivered parenting program may help teens with self-harm. Eligible parents will be assigned by chance (like flipping a coin) to one of two conditions: (1) Healthy Emotions and Relationships with Teens – A Guide for Parents (HEART-P): a 12-session individually-delivered parenting program that teaches parents skills and strategies to help them respond to their adolescents’ emotions, or (2) wait list: parents will be offered the opportunity to receive HEART-P following their completion of the assessments during the study phase.

The purpose of this study is to find out if using a computer program (called iDose) to guide infliximab dosing is more effective and safer than using standard infliximab dosing over 52 weeks. All patients in this study will be receiving infliximab as part of their medical care, this study is only looking at two different methods of determining the dose and timing of administration.

The purpose of this open-label Expanded Access Protocol (EAP) is to provide access to MBP134, for treatment of Sudan Virus Disease (SVD). Patients will receive a single IV infusion of 50 mg/kg MBP134. Patients will be monitored and assessed daily through discharge for safety and the incidence of serious adverse events (SAEs), and of all adverse events (AEs) during infusions.

In the first phase of the study, participants will be asked to complete two sets of appointments six months apart. During both sets of appointments, participants will be asked to complete interviews and questionnaires about their life experiences and mental health, and they will have an EEG and fMRI while completing computerized tasks. The second phase of the study is optional. In this phase, participants will test one of two forms of computerized cognitive training, or brain games. They will be asked to complete 10 hours of training over a 3-6 week period. After the training period is over, they will have two sets of follow up visits, one right after the training period and one five months later. At these appointments, participants will complete the same activities done in the first phase of this study, including the interviews, questionnaires, and imaging (fMRI and EEG) combined with computerized tasks. We are recruiting two groups of participants for this study. One group will include individuals who experience hallucinations, delusions, paranoia, or a psychosis disorder (i.e., schizophrenia), and the other group will be individuals who do not have a diagnosis or family history of schizophrenia, bipolar disorder, or autism spectrum disorder.

The purpose of this research study is to collect timely and relevant data that will support the evolving research needs of the Inherited Retinal Disease community (IRD), in order to provide insights that can be used to improve patient management, and to inform development of future treatments. No visits, assessments, or procedures are mandated, and follow-up will be captured as part of your standard of care. The planned length of registry is of 8 years with a potential to extend the duration as needs evolve.

The purpose of this study is to learn more about the safety and effectiveness of human growth hormone treatments, in routine practice.

Prospective study investigating brain activity associated with markers of suicidal behavior (SB) in adolescents.

This phase I/II trial studies how well tiragolumab and atezolizumab works when given to children and adults with SMARCB1 or SMARCA4 deficient tumors that that has either come back (relapsed) or does not respond to therapy (refractory). SMARCB1 or SMARCA4 deficiency means that tumor cells are missing the SMARCB1 and SMARCA4 genes, which is related to having more aggressive cancers that are harder to treat. Immunotherapy with monoclonal antibodies, such as tiragolumab and atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

This is a double-blind randomized placebo-controlled clinical trial examining choline supplementation in 2-5 year old children with Fetal Alcohol Spectrum Disorders. Outcome measures are neurocognitive tests of memory, attention, and behavior.

We are testing whether treating people who have FSGS with plasmapheresis and rituximab before or shortly after kidney transplant can prevent the recurrence of FSGS after kidney transplant. All participants will receive plasmapheresis. Each participant has a 50% chance of receiving rituximab and a 50% chance of receiving no additional treatment. Rituximab is given by infusion. If a participant is assigned to receive rituximab, it will be given one time immediately after plasmapheresis.

Topiramate is a commonly prescribed medication at weight management clinics. While doctors know that some people respond better to topiramate in terms of weight loss and changes in eating behaviors, the reasons WHY some respond better than others are not known. Knowing more about the relationships between topiramate doses, concentrations of topiramate in the blood stream, and an individual’s response to this medicine will help doctors determine those who may be more likely to benefit. Doctors also want to know if someone’s genes (their DNA) may help explain why some people respond better to topiramate than others. We expect that you will be in this research study for about 14 weeks (3.5 months).

This is a study enrolling patients receiving care at early psychosis specialty coordinated care programs across the United States. Participants will be randomly assigned to one of two groups. The first group will use two mobile apps, computerized brain training and a motivational smart phone app, for a period of 12 weeks. The other group will participate in their regular clinical care. There will be 4 study timepoints: intake, post-training, 6-month follow up, and 12-month follow up. There is also an optional interview about experiences of loneliness.

This is a multicenter, single-arm, open label trial, with iptacopan treatment for 24 weeks in adult PNH patients. Eligible participants must have a mean Hb ≥10 g/dL in response to a stable regimen with anti-C5 for at least 6 months and must be transfusion free for the same period.

A Phase I/II trial of single agent intravenous CBL0137 in pediatric patients (≥ 12 months and ≤ 30 years) with relapsed/refractory solid tumors, including CNS tumors and lymphoma.

This is a study of persons who do and do not stutter between ages 7 and 65 years. This study is trying to figure out how attention skills influence memory, language, and speech fluency skills.

The effect of enzyme replacement therapy on how well your kidneys are responding to enzyme replacement therapy (ERT) is not clear from blood and urine tests alone, but may be more clear in comparisons of kidney biopsies performed before and some time after ERT has been initiated, and this is what we are focusing our study efforts on. The purpose of this study is to obtain your permission to allow us to study the kidney biopsy tissues (collected for medical reasons) after the regular routine studies have been completed. Through our special research measurements and additional study, we hope to be able to see and measure very specific changes in the kidney tissues from Fabry patients taking ERT. We also hope that through these studies of what happens within the kidney before and after starting ERT, we are able to reveal valuable information about the importance of factors like your age that you started ERT, the amount or dosage of ERT, and any differences seen between males and females.

The purpose of this study is to determine the dose of medication (Valacyclovir) needed to prevent an infant from developing herpes simplex virus (HSV) if the infant was potentially exposed to HSV at the time of delivery as they passed through the birth canal.

The primary study objective is to determine whether “Take on Transplant” (ToT), a CF-specific Lung Transplant (LTx) educational website, improves patient-reported preparedness for LTx discussions, as measured by the Preparation for Decision Making (PrepDM) Scale at 3 months after randomization, compared to an attention control transplant website (unos.org, UNOS).

The research aspect of this trial is the use of a new machine to remove specific lymphocytes from the donor’s peripheral blood stem cells (PBSCs). This is called T cell receptor alpha/beta T cell depletion. This machine does such a good job at removing the unwanted donor T cells, and as a result we think patients will need fewer drugs to suppress their immune system.

This research trial studies the Project: Every Child for younger patients with cancer. Gathering health information over time from younger patients with cancer may help doctors find better methods of treatment and on-going care.

This is an open-label, multi-center, Phase 1/2 study of oral LOXO-292 in pediatric patients with an activating RET alteration and an advanced solid or primary CNS tumor.

This research is being done to help better understand the ways parents interact with their children at meal and snack times at different time points during the day and in different circumstances. We want to study how the feeding approaches affect the child’s dietary intake over time. Study participation will be two years with activities every six months.